This is an open label, single arm, phase 2 trial investigating bezuclastinib plus sunitinib in patients with GIST who have previously progressed on sunitinib.

开始日期2024-08-01

申办/合作机构

Sarcoma Alliance for Research through Collaboration

[+3]

NCT05186753

/ Active, not recruiting临床2期

A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of The Safety and Efficacy of CGT9486 in Subjects With Nonadvanced Systemic Mastocytosis

This is a multi-part, randomized, double-blind, placebo-controlled Phase 2 clinical study comparing the safety and efficacy of bezuclastinib (CGT9486) plus best supportive care (BSC) with placebo plus BSC in patients with nonadvanced systemic mastocytosis (NonAdvSM), including indolent systemic mastocytosis and smoldering systemic mastocytosis, whose symptoms are not adequately controlled by BSC. This study will be conducted in three parts. Patients in Parts 1a, 1b and 2 will receive bezuclastinib or placebo, and may roll over onto Part 3 to receive treatment with bezuclastinib. Additionally, a substudy of subjects will investigate the efficacy, safety, and tolerability of bezuclastinib in patients who are experiencing inadequate symptom control with avapritinib.

开始日期2022-06-27

申办/合作机构

Cogent Biosciences, Inc.

NCT05208047

/ Active, not recruiting临床3期

A Phase 3 Randomized, Open-Label, Multicenter Clinical Study of CGT9486+Sunitinib vs. Sunitinib in Subjects With Locally Advanced, Unresectable, or Metastatic Gastrointestinal Stromal Tumors

This is a Phase 3, open-label, international, multicenter study of CGT9486 in combination with sunitinib. This is a multi-part study that will enroll approximately 442 patients. Part 1 consists of two evaluations: 1) confirming the dose of an updated formulation of CGT9486 to be used in subsequent parts in approximately 20 patients who have received at least one prior line of therapy for GIST and 2) evaluating the potential for drug-drug interactions between CGT9486 and sunitinib in approximately 18 patients who have received at least two prior tyrosine kinase inhibitors (TKIs) for GISTs. The second part of the study will enroll approximately 388 patients who are intolerant to, or who failed prior treatment with imatinib only and will compare the efficacy of CGT9486 plus sunitinib to sunitinib alone with patients being randomized in a 1:1 manner. Additionally, a drug-drug interactions substudy will investigate the potential for CGT9486 to be a CYP3A4 inducer in approximately 16 patients who have received at least one prior line of therapy for GIST.

开始日期2022-04-14

申办/合作机构

Cogent Biosciences, Inc.

100 项与贝祖克拉替尼相关的临床结果

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100 项与贝祖克拉替尼相关的专利（医药）

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项与贝祖克拉替尼相关的文献（医药）

2023-07-03·Expert opinion on emerging drugs

Systemic mastocytosis: 2023 update on diagnosis and management in adults

Review

作者: Costa, Alessandro ; Masucci, Chiara ; Carmosino, Ida ; Ielo, Claudia ; Capriata, Marcello ; Passucci, Mauro ; Scalzulli, Emilia ; Martelli, Maurizio ; Breccia, Massimo

INTRODUCTION:

Systemic mastocytosis (SM) is a complex and heterogeneous disease, characterized by the clonal accumulation of mast cells in one or more organs. In 2022 both the World Health Organization (WHO) and the International Consensus Classification (ICC) modified the diagnostic and classification criteria of SM. Moreover, the identification of new clinical and molecular variables has improved prognostic tools and led to increasingly individualized therapeutic strategies.

AREAS COVERED:

The aim of this review is to present the updates introduced by the International Consensus Classification in diagnostic criteria of SM. In addition, we report the latest data available from the most important clinical trials in patients both with non-advanced and advanced disease, including elenestinib and bezuclastinib.

EXPERT OPINION:

Diagnosis and classification of SM has evolved over years. The most recent WHO and ICC classification improved SM diagnostic work-up, providing clinicians with a clear and simplified diagnostic scheme. New approved targeted therapies such as midostaurin and avapritinib modified the treatment paradigm in patients in advanced stage, and next-generation inhibitors actually investigated in clinical trials are expected in the next future.

2022-12-09·Hematology. American Society of Hematology. Education Program

Available and emerging therapies for bona fide advanced systemic mastocytosis and primary eosinophilic neoplasms

Review

作者: Gotlib, Jason

Abstract:

The historically poor prognosis of patients with advanced systemic mastocytosis (AdvSM) and primary eosinophilic neoplasms has shifted to increasingly favorable outcomes with the discovery of druggable targets. The multikinase/KIT inhibitor midostaurin and the highly selective KIT D816V inhibitor avapritinib can elicit marked improvements in measures of mast cell (MC) burden as well as reversion of MC-mediated organ damage (C-findings) and disease symptoms. With avapritinib, the achievement of molecular remission of KIT D816V and improved survival compared with historical therapy suggests a potential to affect disease natural history. BLU-263 and bezuclastinib are KIT D816V inhibitors currently being tested in trials of AdvSM. In the new World Health Organization and International Consensus Classifications, the category of “myeloid/lymphoid neoplasms with eosinophilia and tyrosine kinase (TK) gene fusions” is inclusive of rearrangements involving PDGFRA, PDGFRB, FGFR1, JAK2, FLT3, and ETV6::ABL1. While the successful outcomes with imatinib in FIP1L1::PDGFRA-positive cases and PDGFRB-rearranged neoplasms have become the “poster children” of these disorders, the responses of the other TK-driven neoplasms to small-molecule inhibitors are more variable. The selective FGFR inhibitor pemigatinib, approved in August 2022, is a promising therapy in aggressive FGFR1-driven diseases and highlights the role of such agents in bridging patients to allogeneic transplantation. This review summarizes the data for these approved and investigational agents and discusses open questions and future priorities regarding the management of these rare diseases.

2021-09-01·JAMA oncology1区 · 医学

Association of Combination of Conformation-Specific KIT Inhibitors With Clinical Benefit in Patients With Refractory Gastrointestinal Stromal Tumors

1区 · 医学

Article

作者: Tinoco, Gabriel ; Sanftner, Laura ; Tsang, Garson ; Habets, Gaston ; Shields, Anthony F. ; Alcantar, Oscar ; Shellooe, Rafe ; Zhang, Chao ; Wagner, Andrew J. ; Tsai, James ; Trent, Jonathan C. ; Tsiatis, Athanasios C. ; Carias, Heidi ; Zhang, Ying ; Chan, Katrina ; Ewing, Todd ; Spevak, Wayne ; Lin, Jack ; Tap, William D. ; Inokuchi, Kerry ; Burton, Elizabeth A. ; Bollag, Gideon ; Nespi, Marika ; Michelson, Glenn ; Patnaik, Amita ; Ibrahim, Prabha N. ; Severson, Paul L. ; Chugh, Rashmi ; Matusow, Bernice ; Rezaei, Hamid ; Marimuthu, Adhirai ; Wu, Guoxian

IMPORTANCE:

Many cancer subtypes, including KIT-mutant gastrointestinal stromal tumors (GISTs), are driven by activating mutations in tyrosine kinases and may initially respond to kinase inhibitors but frequently relapse owing to outgrowth of heterogeneous subclones with resistance mutations. KIT inhibitors commonly used to treat GIST (eg, imatinib and sunitinib) are inactive-state (type II) inhibitors.

OBJECTIVE:

To assess whether combining a type II KIT inhibitor with a conformation-complementary, active-state (type I) KIT inhibitor is associated with broad mutation coverage and global disease control.

DESIGN, SETTING, AND PARTICIPANTS:

A highly selective type I inhibitor of KIT, PLX9486, was tested in a 2-part phase 1b/2a trial. Part 1 (dose escalation) evaluated PLX9486 monotherapy in patients with solid tumors. Part 2e (extension) evaluated PLX9486-sunitinib combination in patients with GIST. Patients were enrolled from March 2015 through February 2019; data analysis was performed from May 2020 through July 2020.

INTERVENTIONS:

Participants received 250, 350, 500, and 1000 mg of PLX9486 alone (part 1) or 500 and 1000 mg of PLX9486 together with 25 or 37.5 mg of sunitinib (part 2e) continuously in 28-day dosing cycles until disease progression, treatment discontinuation, or withdrawal.

MAIN OUTCOMES AND MEASURES:

Pharmacokinetics, safety, and tumor responses were assessed. Clinical efficacy end points (progression-free survival and clinical benefit rate) were supplemented with longitudinal monitoring of KIT mutations in circulating tumor DNA.

RESULTS:

A total of 39 PLX9486-naive patients (median age, 57 years [range, 39-79 years]; 22 men [56.4%]; 35 [89.7%] with refractory GIST) were enrolled in the dose escalation and extension parts. The recommended phase 2 dose of PLX9486 was 1000 mg daily. At this dose, PLX9486 could be safely combined with 25 or 37.5 mg daily of sunitinib continuously. Patients with GIST who received PLX9486 at a dose of 500 mg or less, at the recommended phase 2 dose, and with sunitinib had median (95% CI) progression-free survivals of 1.74 (1.54-1.84), 5.75 (0.99-11.0), and 12.1 (1.34-NA) months and clinical benefit rates (95% CI) of 14% (0%-58%), 50% (21%-79%), and 80% (52%-96%), respectively.

CONCLUSIONS AND RELEVANCE:

In this phase 1b/2a nonrandomized clinical trial, type I and type II KIT inhibitors PLX9486 and sunitinib were safely coadministered at the recommended dose of both single agents in patients with refractory GIST. Results suggest that cotargeting 2 complementary conformational states of the same kinase was associated with clinical benefit with an acceptable safety profile.

TRIAL REGISTRATION:

ClinicalTrials.gov Identifier: NCT02401815.

152

项与贝祖克拉替尼相关的新闻（医药）

2026-03-17

·药明康德

产业新闻 | 美国FDA受理小分子新药申请；7750万美元A轮融资！新锐推进潜在“first-in-class”疗法……

美国FDA受理小分子新药申请 Cogent Biosciences近日宣布，美国FDA已受理其bezuclastinib用于治疗非进展性系统性肥大细胞增多症（NonAdvSM）的新药申请（NDA），并将PDUFA目标审评日期设定为2026年12月30日。该申请基于关键性SUMMIT临床试验结果。数据显示，在NonAdvSM患者中，bezuclastinib在主要终点及所有关键次要终点上均实现具有临床意义且高度统计学显著的改善。随访至48周的结果进一步显示，患者症状改善呈持续加深趋势，表明该疗法在更长期用药中具备维持疗效的潜力。此外，该药物在骨密度改善及疾病修饰方面提供了初步证据。安全性方面，SUMMIT研究结果显示bezuclastinib具有良好的安全性和耐受性特征，支持其在该慢性疾病中的长期用药前景。除NonAdvSM适应症外，bezuclastinib在其他适应症中的开发亦稳步推进。公司预计将于2026年4月完成该药物在既往接受过伊马替尼治疗的胃肠道间质瘤（GIST）患者中的新药申请递交，该适应症曾获得突破性疗法认定。同时，bezuclastinib用于治疗进展性系统性肥大细胞增多症（AdvSM）的新药申请也计划于2026年上半年提交。 Bezuclastinib是一种选择性酪氨酸激酶抑制剂，旨在强效抑制KIT基因中关键驱动突变产生的突变蛋白活性。其中一种突变KIT D816V会驱动系统性肥大细胞增多症，这是一种由肥大细胞失控增殖引起的严重疾病。其他KIT突变，包括发生在第17外显子上的多种突变，则会驱动GIST患者的肿瘤生长。 7750万美元A轮融资！新锐推进潜在“first-in-class”疗法今日，R1 Therapeutics宣布正式成立，并完成超额认购的7750万美元A轮融资，同时获得来自Alebund Pharmaceuticals的独家全球许可（不含大中华区），用于开发和商业化候选药物AP306。本轮融资由Abingworth、F-Prime和DaVita Venture Group共同领投，Curie.Bio、SymBiosis及U.S. Renal Care等机构参与投资。公司表示，此次融资所得将用于推进AP306的全球开发计划，并与Alebund开展合作，计划于今年晚些时候启动一项2b期临床研究。 AP306是一种潜在“first-in-class”的泛磷酸盐转运蛋白抑制剂，正在开发作为单药疗法用于治疗慢性肾病（CKD）患者的高磷血症。与当前已获批的降磷疗法主要通过结合磷或抑制被动转运机制不同，AP306的差异化在于其能够阻断磷的主动转运过程，从而提供新的治疗路径。该药物此前已在透析患者中完成2a期临床研究，结果发表于Kidney International Reports期刊当中，显示其在降低血清磷水平方面具有显著疗效，并具备良好的安全性和耐受性。创新庞贝病siRNA疗法临床积极结果公布 Aro Biotherapeutics今日公布其在研siRNA疗法ABX1100用于治疗晚发型庞贝病（LOPD）的1b期部分临床试验积极顶线结果。研究显示，ABX1100能够实现对糖原合酶1（GYS1）mRNA的强效且持续性下调，该酶是肌肉中糖原生成的关键调控因子。与此同时，试验还观察到患者肺功能及探索性生物标志物的改善，提示该疗法可能带来功能性获益。在9例LOPD患者中，ABX1100总体耐受性良好，安全性结果与此前在29名健康志愿者中开展的1a期研究一致。在疗效方面，研究通过预测用力肺活量百分比（FVC %pred）评估肺功能变化。与基线相比，接受治疗的7名患者在5个月时FVC平均提升约2.5%，其中中重度患者的改善更为显著，提示较快的功能应答。此外，在第10周时，多数患者的肌酸激酶（CK）和尿液Hex4等探索性生物标志物较基线下降，与GYS1 mRNA下调及潜在功能改善相一致。安全性方面，研究未观察到输注反应、严重不良事件或因治疗中断的情况，也未发现具有临床意义的实验室异常。ABX1100由可结合CD71受体的Centyrin与siRNA偶联构成，通过特异性抑制GYS1表达以降低肌肉中糖原生成水平，目前已获得美国FDA孤儿药资格和罕见儿科疾病认定。参考资料： [1] Cogent Biosciences Announces FDA Acceptance of New Drug Application for Bezuclastinib in Patients with NonAdvanced Systemic Mastocytosis (NonAdvSM). Retrieved March 17, 2026 from https://www.globenewswire.com/news-release/2026/03/16/3256200/0/en/Cogent-Biosciences-Announces-FDA-Acceptance-of-New-Drug-Application-for-Bezuclastinib-in-Patients-with-NonAdvanced-Systemic-Mastocytosis-NonAdvSM.html [2] R1 Therapeutics launches with oversubscribed $77.5 million Series A financing to advance first-in-class treatment for hyperphosphatemia in patients with chronic kidney disease. Retrieved March 17, 2026 from https://www.globenewswire.com/news-release/2026/03/17/3256979/0/en/R1-Therapeutics-launches-with-oversubscribed-77-5-million-Series-A-financing-to-advance-first-in-class-treatment-for-hyperphosphatemia-in-patients-with-chronic-kidney-disease.html [3] Aro Biotherapeutics Reports Positive Phase 1b Topline Results for ABX1100, a Muscle-targeted GYS1 siRNA, in Patients with Late-Onset Pompe Disease (LOPD). Retrieved March 17, 2026 from https://www.businesswire.com/news/home/20260316177283/en/Aro-Biotherapeutics-Reports-Positive-Phase-1b-Topline-Results-for-ABX1100-a-Muscle-targeted-GYS1-siRNA-in-Patients-with-Late-Onset-Pompe-Disease-LOPD 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

siRNA突破性疗法临床2期

2026-03-16

·BioSpace

Cogent Biosciences Announces FDA Acceptance of New Drug Application for Bezuclastinib in Patients with NonAdvanced Systemic Mastocytosis (NonAdvSM)

NDA based on positive clinical results from the SUMMIT pivotal trial in which bezuclastinib demonstrated clear clinical benefit across all symptom domains WALTHAM, Mass. and BOULDER, Colo., March 16, 2026 (GLOBE NEWSWIRE) -- Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for bezuclastinib in patients with NonAdvanced Systemic Mastocytosis (NonAdvSM) and assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2026. In addition, the FDA communicated that at this time, there is no plan to hold an advisory committee, nor have they identified any potential review issues. Clinical results from the pivotal SUMMIT trial with bezuclastinib in NonAdvSM patients demonstrated clinically meaningful and highly statistically significant improvements across the primary and all key secondary endpoints. Data from the SUMMIT trial through 48 weeks showcased a clear and continued deepening of symptomatic improvement over time, supporting the potential for sustained clinical benefit with longer duration of therapy. In addition, results also showed the benefit of bezuclastinib in populations with high unmet need and bezuclastinib’s impact on bone mineral density as well as evidence of disease modification. Across the SUMMIT trial, bezuclastinib demonstrated a favorable safety and tolerability profile, supporting its potential for chronic use in patients with NonAdvSM. Completion of the NDA submission for bezuclastinib in patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib remains on track for April 2026. The GIST submission was initiated under Real-Time Oncology Review (RTOR), and bezuclastinib received Breakthrough Therapy Designation in this indication. The NDA submission for bezuclastinib in patients with Advanced Systemic Mastocytosis (AdvSM) remains on track for the first half of 2026. About Cogent Biosciences, Inc. Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases. The most advanced clinical program, bezuclastinib, is a selective tyrosine kinase inhibitor that is designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17. KIT D816V is responsible for driving systemic mastocytosis, a serious disease caused by unchecked proliferation of mast cells. Exon 17 mutations are also found in patients with advanced gastrointestinal stromal tumors (GIST), a type of cancer with strong dependence on oncogenic KIT signaling. In addition, the Cogent Research Team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases targeting mutations in ErbB2, PI3Kα, KRAS and JAK2. Cogent Biosciences is based in Waltham, MA and Boulder, CO. Visit our website for more information at  . Follow Cogent Biosciences on social media:  X  (formerly known as Twitter) and  LinkedIn . Information that may be important to investors will be routinely posted on our website and  X . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding: the potential for bezuclastinib to deliver sustained clinical benefit with longer duration of therapy for patients with NonAdvSM; the potential for bezuclastinib’s chronic use in patients with NonAdvSM; the company’s expectation that it will complete its NDA submission for bezuclastinib in patients with GIST in April 2026; and the company’s expectation that it will submit its NDA for bezuclastinib in patients with AdvSM in the first half of 2026. The use of words such as, but not limited to, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "will," or "would" and similar words expressions are intended to identify forward-looking statements. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, our clinical results, the rate of enrollment in our clinical trials and other future conditions. New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. We may not actually achieve the forecasts or milestones disclosed in our forward-looking statements, and you should not place undue reliance on our forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to those set forth under the caption "Risk Factors" in Cogent's most recent Quarterly Report on Form 10-Q filed with the SEC. Any forward-looking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors or representatives, undertake any obligation to publicly update or revise any forward-looking statement, whether as result of new information, future events or otherwise, except as required by law. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date hereof. Contact: Christi Waarich Senior Director, Investor Relations christi.waarich@cogentbio.com 617-830-1653

申请上市临床结果突破性疗法优先审批

2026-03-14

·思索新知

罕见病药物市场将突破4000亿美元，小分子药物正在重新崛起

过去几年，很多人一提到罕见病治疗，第一反应往往是基因治疗、RNA疗法或者细胞疗法。但如果把整个研发管线放在一起仔细看，会发现一个有意思的变化：传统的小分子药物，正在重新回到罕见病研发的舞台中央。一份最新行业报告给出了一个非常直观的数据。到2032年，全球罕见病药物销售额预计将超过4000亿美元，而2025年的市场规模大约是2130亿美元。换句话说，未来几年罕见病市场仍然会保持相当快速的增长。如果这个预测实现，罕见病药物在2032年将占到全球制药市场销售额的五分之一以上。这意味着，在整个医药产业中，罕见病治疗正在变成越来越重要的一块。更有意思的是，在目前最有商业潜力的罕见病研发管线中，小分子药物占据了非常大的比例。根据Evaluate的分析，在目前价值最高的20个罕见病在研药物中，大约45%属于小分子药物。这个比例明显高于单克隆抗体等生物药。后者只占大约20%，而DNA、RNA疗法以及细胞和基因治疗加起来的比例反而更小。从某种意义上说，这个结果有点出人意料。因为在过去几年里，公众讨论最多的往往是基因治疗、CRISPR编辑或者RNA疗法，但真正处在研发管线核心位置的，依然有相当一部分是传统的小分子药物。事实上，在目前上市销售额最高的孤儿药中，小分子药物的数量并不多。报告中提到，目前最畅销的孤儿药里，小分子药物主要只有三个代表，包括Vertex开发的囊性纤维化药物Alfytrek和Trikafta，以及BeOne用于血液肿瘤治疗的Brukinsa。但在研发阶段，小分子药物却明显更活跃。在报告列出的重点候选药物中，包括Revolution Medicines开发的pan-RAS抑制剂daraxonrasib，用于胰腺肿瘤；Priovant开发的TYK2/JAK1双重抑制剂brepocitinib，用于自身免疫疾病；以及Cogent Biosciences开发的bezuclastinib，用于系统性肥大细胞增多症。这些项目的共同特点是，它们都属于精准靶向的小分子药物，通过直接作用于特定信号通路或蛋白靶点来发挥作用。小分子药物的“第二次机会” 为什么在基因治疗和RNA技术不断发展的今天，小分子药物反而重新受到关注？一个重要原因是，科学界对疾病机制的理解正在不断加深。随着基因组学、蛋白组学以及AI辅助药物设计技术的发展，研究人员能够更精准地找到新的治疗靶点。与此同时，化学合成技术和数据分析工具也在不断进步，让小分子药物的设计效率明显提高。除了技术进步之外，小分子药物本身也有一些非常现实的优势。比如它们通常可以口服使用，生产成本相对较低，药物稳定性也更好。这些特点在长期治疗的慢性罕见病中尤其重要。当然，小分子药物的发展也并不是没有挑战。在美国，新的药品定价政策正在对不同类型药物产生不同影响。根据《通胀削减法案》（Inflation Reduction Act），小分子药物在上市9年后就可能面临政府价格谈判，而生物药则可以获得13年的保护期。这种差异被很多药企称为“pill penalty”，因为它在一定程度上降低了企业开发小分子药物的积极性。与此同时，罕见病药物研发本身也正在经历一段比较复杂的时期。一方面，监管机构在审批标准上变得越来越严格。最近几年，多个罕见病药物项目在FDA审批阶段遇到挫折，一些公司不得不调整研发计划，甚至重新设计临床试验。例如uniQure开发的亨廷顿病基因治疗AMT-130，就曾在监管沟通中经历政策变化。此前FDA一度认可公司提出的临床研究方案，但后来又改变了态度，不再接受外部对照研究作为审批依据。类似的情况也出现在其他罕见病公司身上，包括Capricor和Biohaven等企业，都因为监管要求的变化而不得不调整开发时间表，甚至重新规划公司战略。监管环境的不确定性，再加上资金压力，使得一些罕见病研发项目被迫暂停甚至终止。但即便如此，整个罕见病市场的长期增长趋势仍然非常明显。报告认为，随着越来越多精准治疗技术进入临床阶段，罕见病药物市场在未来几年仍将持续扩大。到2032年，超过4000亿美元的市场规模将使其成为制药行业最重要的增长领域之一。与此同时，一些政策层面的利好也正在出现。例如，美国重新恢复了“罕见儿科疾病优先审评券”（rare pediatric disease priority review voucher）计划，并将该项目延续到2029年。这个机制允许开发罕见儿科疾病治疗的公司获得一张优先审评券，这张券可以用于加速其他药物审批，甚至可以在市场上出售，因此对企业具有很高的商业价值。在多种技术路线同时发展的今天，罕见病治疗已经不再只有单一方向。基因治疗、RNA疗法、细胞治疗以及小分子药物，正在共同构成一个越来越多元的治疗生态。而从当前研发管线的情况来看，小分子药物很可能正在迎来一次新的发展周期。 🔹本文供医学科普使用，不构成医疗建议。本文内容基于公开资料整理，包括但不限于Fierce Biotech、The Guardian、Reuters、Nature、STAT News 等权威媒体与期刊。所有翻译与解读仅用于知识分享与医学科普，不构成任何医疗建议或临床指导。 🔹本文由「思索新知」团队基于海外权威资料整理，致力于提供可信赖的全球医疗科技动态。

100 项与贝祖克拉替尼相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
系统性肥大细胞增多症	申请上市	美国	Cogent Biosciences, Inc.	2025-12-30
转移性胃肠道间质瘤	临床3期	美国	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	阿根廷	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	澳大利亚	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	巴西	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	加拿大	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	智利	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	捷克	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	丹麦	Cogent Biosciences, Inc.	2022-04-14
转移性胃肠道间质瘤	临床3期	法国	Cogent Biosciences, Inc.	2022-04-14

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05186753 (Summit, ASH2025) 人工标引	临床2期	系统性肥大细胞增多症 KIT c.2447 C>T (p.D816V) mutation	179	Bezuclastinib + BSC	鑰繭鑰鏇醖窪網觸襯簾(憲壓憲蓋觸齋願選選鑰) = 範憲窪觸壓蓋鹽遞積築鹽選鏇遞觸鹽憲願廠醖 (膚鹽構構選觸膚鹹築顧, -27.6 ~ -21.1) 更多	积极	2025-12-06
				Placebo + BSC	鑰繭鑰鏇醖窪網觸襯簾(憲壓憲蓋觸齋願選選鑰) = 選廠鹽繭獵糧淵壓繭襯鹽選鏇遞觸鹽憲願廠醖 (膚鹽構構選觸膚鹹築顧, -19.6 ~ -11.2) 更多
NCT05208047 (PEAK, Company_Website) 人工标引	临床3期	胃肠道间质瘤二线	-	bezuclastinib + sunitinib	壓夢獵衊積繭餘壓願壓(衊鹽齋遞襯繭憲艱憲範) = 範膚糧醖壓壓範夢繭醖餘夢壓膚獵築糧憲糧願 (膚鑰獵網蓋淵鹹鹹遞獵 ) 更多	积极	2025-11-10
				sunitinib	壓夢獵衊積繭餘壓願壓(衊鹽齋遞襯繭憲艱憲範) = 蓋鬱製積艱淵窪襯顧憲餘夢壓膚獵築糧憲糧願 (膚鑰獵網蓋淵鹹鹹遞獵 ) 更多
NCT05186753 (SUMMIT, Corporate Publications 1 \| Corporate Publications 2 \| Company_Website) 人工标引	临床2期	系统性肥大细胞增多症 \| 肥大细胞增多症	179	Bezuclastinib	觸鬱網鬱築鏇艱顧觸憲(築糧齋顧襯襯願憲壓膚) = 鏇齋膚廠憲網獵壓鏇窪鑰願襯廠願鹹醖遞鹹願 (壓襯餘艱醖構遞夢顧鏇 ) 达到更多	积极	2025-07-07
				placebo	觸鬱網鬱築鏇艱顧觸憲(築糧齋顧襯襯願憲壓膚) = 衊壓簾顧艱鏇鹽觸簾網鑰願襯廠願鹹醖遞鹹願 (壓襯餘艱醖構遞夢顧鏇 ) 达到更多
NCT06208748 (SARC044, ASCO2025)	临床2期	胃肠道间质瘤 KIT exon 9	40	Bezuclastinib 600 mg daily + Sunitinib 37.5 mg daily	夢願築淵壓積範製衊齋(窪廠鹹齋獵築憲艱鏇獵) = 餘顧糧襯膚醖範積鬱夢窪憲壓簾壓醖範簾夢簾 (繭衊選鏇鏇願糧積廠艱 )	积极	2025-05-30
NCT05186753 (SUMMIT, Company_Website) 人工标引	临床2期	系统性肥大细胞增多症	-	Bezuclastinib 100 mg	鏇顧齋襯齋觸繭願遞齋(憲觸鬱鹹窪簾壓鏇觸糧) = 糧構鹽積鹹衊艱夢築襯構繭築範廠構鑰膚鏇齋 (繭憲鹹餘鹽憲蓋顧製顧 ) 更多	积极	2025-02-27
				Placebo	-
NCT05186753 (SUMMIT, GlobeNewswire) 人工标引	N/A	系统性肥大细胞增多症	27	Bezuclastinib 100 mg	艱憲遞糧窪顧築餘選製(夢壓遞鏇蓋壓築齋製淵) = 顧憲廠壓遞蓋願糧觸觸鬱鹹糧鑰獵窪鏇願襯鏇 (築網壓遞廠範襯觸築構 ) 更多	积极	2024-12-09
NCT04996875 (Apex, ASH2024) 人工标引	临床2期	系统性肥大细胞增多症	32	Bezuclastinib	蓋襯醖餘窪鏇鬱築繭憲(鹽選蓋網廠鬱糧選築網) = 繭鏇鬱構糧蓋淵築鬱衊醖鹽醖膚構壓繭齋醖鹽 (膚膚襯鹽鏇鑰夢鑰艱廠 ) 更多	积极	2024-12-08
NCT05186753 (Summit, SOHO2024)	临床2期	侵袭性系统性肥大细胞增多症 Serum tryptase \| KIT D816V positive	-	Bezuclastinib low dose	網廠願積願遞餘餘選鏇(築餘膚膚餘鹹鹹構膚顧) = 網獵鹹衊獵獵廠鏇膚鹽壓製製衊網鑰襯繭遞廠 (鏇顧簾鏇鹹遞繭衊糧餘 ) 更多	积极	2024-09-04
				Bezuclastinib high dose	網廠願積願遞餘餘選鏇(築餘膚膚餘鹹鹹構膚顧) = 艱壓築遞憲網窪獵衊襯壓製製衊網鑰襯繭遞廠 (鏇顧簾鏇鹹遞繭衊糧餘 ) 更多
NCT05208047 (Peak, ASCO2024) 人工标引	临床3期	胃肠道间质瘤 KIT Mutation	42	Bezuclastinib	醖鬱壓醖鬱遞齋繭獵憲(築觸淵獵觸糧鑰繭齋糧) = The majority of TEAEs were low grade and reversible with low rate (38%) of Grade 3+ events 積糧夢壓憲鬱衊襯範醖 (衊齋醖願夢窪艱網構壓 )	积极	2024-05-24
NCT05186753 (Summit, AAAAI2024) 人工标引	临床2期	系统性肥大细胞增多症 KIT D816V positive	50	Bezuclastinib low dose	鹹夢選願壓獵鬱餘窪觸(鏇衊窪選壓憲窪獵糧繭) = 鹽網艱鹽鹽願糧繭壓鹹顧願醖窪襯窪範鑰遞窪 (膚廠遞鑰網鬱製積淵廠 ) 更多	积极	2024-02-23
				Bezuclastinib high dose	鹹夢選願壓獵鬱餘窪觸(鏇衊窪選壓憲窪獵糧繭) = 鑰繭窪鏇範壓鹽艱醖蓋顧願醖窪襯窪範鑰遞窪 (膚廠遞鑰網鬱製積淵廠 ) 更多